DelveInsight’s “Duchenne Muscular Dystrophy Pipeline Insight 2025” report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in the Duchenne Muscular Dystrophy pipeline landscape. It covers the Duchenne Muscular Dystrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Duchenne Muscular Dystrophy therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Duchenne Muscular Dystrophy Pipeline Report

• In May 2025, Edgewise Therapeutics Inc. announced a EDG-5506-201 CANYON study was expanded to include an additional 120 adult participants in a cohort called GRAND CANYON, that is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of sevasemten in adults with Becker.
• In May 2025, REGENXBIO Inc. conducted a phase I/II/III study to evaluate the safety, tolerability, pharmacodynamics (microdystrophin protein levels), pharmacokinetic, and clinical efficacy of RGX-202 when administered IV as one-time dose to ambulant male participants with Duchenne. A comprehensive, short-term, prophylactic immunosuppression regimen will be administered during treatment to mitigate a potential immune response.
• In May 2025, Italfarmaco organized a study will evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant patients to further corroborate data from the completed phase 3 pivotal study of givinostat in ambulant patients with DMD (ie, Study DSC/14/2357/48, NCT02851797). Primary Objective of the study is to demonstrate the efficacy of givinostat in reducing muscle decline in non-ambulant DMD patients, as measured by Performance of the Upper Limb (PUL) 2.0.
• In May 2025, Pfizer announced a study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies. We will follow participants’ experience in this study for 10 years after the end of their previous study. Participants will have 1 annual onsite visit and a few annual remote visits. The exact number of remote visits will be decided by their study doctor.
• In May 2025, Hoffmann-La Roche conducted a study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
• DelveInsight’s Duchenne Muscular Dystrophy pipeline report depicts a robust space with 75+ active players working to develop 75+ pipeline therapies for Duchenne Muscular Dystrophy treatment.
• The leading Duchenne Muscular Dystrophy Companies such as Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, EDG 5506 Edgewise Therapeutics, Fordadistrogene movaparvovec, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, and others.
• Promising Duchenne Muscular Dystrophy Therapies such as Vamorolone, Sevasemten 10 mg, Givinostat, DS-5141b, SGT-003, PF-06939926, NS-089/NCNP-02, and others.

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Duchenne Muscular Dystrophy Emerging Drugs

• Vamorolone: Santhera

Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors1,2. This has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore could emerge as a valuable alternative to corticosteroids, the current standard of care in children and adolescent patients with DMD. There is a clear unmet medical need in this patient group as high dose corticosteroids have significant systemic side effects that detract from patient quality of life. On September 2, 2020, Santhera exercised its option and obtained worldwide rights to vamorolone in Duchenne muscular dystrophy and all other indications. Santhera and ReveraGen expect to complete the rolling NDA submission to the U.S. FDA in June 2022.

• Givinostat: Italfarmaco

Givinostat, is an HDAC inhibitor (HDACi, a principle candidate, currently being developed for the treatment of DMD and BMD. Since Givinostat acts on the pathogenetic events downstream of the genetic defects, it is potentially a treatment for the whole DMD and BMD population and to counter the disease pathogenetic events in all muscular districts.

• Pamrevlumab: Fibrogen

Pamrevlumab is a first-in-class antibody developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Pamrevlumab is advancing towards Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and pancreatic cancer and has been granted Orphan Drug Designation (ODD) in each of these indications, and is currently in a Phase 2 trial for Duchenne muscular dystrophy (DMD).

The Duchenne Muscular Dystrophy Pipeline Report Provides Insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Duchenne Muscular Dystrophy with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Duchenne Muscular Dystrophy Treatment.
• Duchenne Muscular Dystrophy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Duchenne Muscular Dystrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Duchenne Muscular Dystrophy market.

Learn more about Duchenne Muscular Dystrophy Drugs opportunities in our groundbreaking Duchenne Muscular Dystrophy Research and development projects @ Duchenne Muscular Dystrophy Unmet Needs

Duchenne Muscular Dystrophy Companies

Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, EDG 5506 Edgewise Therapeutics, Fordadistrogene movaparvovec, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, and others.

Duchenne Muscular Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration

• Oral
• Intravenous
• Subcutaneous

Duchenne Muscular Dystrophy Products have been categorized under various Molecule types such as

• Small molecule
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy

Discover the latest advancements in Duchenne Muscular Dystrophy Treatment by visiting our website. Stay informed about how we’re transforming the future of musculoskeletal @ Duchenne Muscular Dystrophy Market Drivers and Barriers, and Future Perspectives

Scope of the Duchenne Muscular Dystrophy Pipeline Report

• Coverage- Global
• Duchenne Muscular Dystrophy Companies- Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, EDG 5506 Edgewise Therapeutics, Fordadistrogene movaparvovec, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, and others.
• Duchenne Muscular Dystrophy Therapies- Vamorolone, Sevasemten 10 mg, Givinostat, DS-5141b, SGT-003, PF-06939926, NS-089/NCNP-02, and others.
• Duchenne Muscular Dystrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Duchenne Muscular Dystrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

For a detailed overview of our latest research findings and future plans, read the full details of Duchenne Muscular Dystrophy Pipeline on our website @ Duchenne Muscular Dystrophy Drugs and Companies

Table of Contents

1. Introduction
2. Executive Summary
3. Duchenne Muscular Dystrophy: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Duchenne Muscular Dystrophy– DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Delandistrogene moxeparvovec: Roche
9. Drug profiles in the detailed report…..
10. Mid-Stage Products (Phase II)
11. SRP 5051: Sarepta Therapeutics
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I/II)
14. WVE N531: Wave Life Sciences
15. Drug profiles in the detailed report…..
16. Early Stage Products (Phase I)
17. EDG 5506: Edgewise Therapeutics
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Duchenne Muscular Dystrophy Key Companies
21. Duchenne Muscular Dystrophy Key Products
22. Duchenne Muscular Dystrophy- Unmet Needs
23. Duchenne Muscular Dystrophy- Market Drivers and Barriers
24. Duchenne Muscular Dystrophy- Future Perspectives and Conclusion
25. Duchenne Muscular Dystrophy Analyst Views
26. Duchenne Muscular Dystrophy Key Companies
27. Appendix

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